Date post: | 14-Dec-2014 |
Category: |
Business |
Upload: | conferenceforum |
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Development Process: Plan with the End in Mind
Protocols
Protocol Outlines
Clinical Development Plan
Non-Clinical Plan
Commercialization
NDA (BLA)/MAA Submission(s)
Registration Strategy
Integrated Summaries
Final Reports
Study Conduct
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Drug Development Regulatory Provisions
§ Fast Track Development Status - To expedite product development
§ Orphan Drug Status - To encourage development of drugs for rare diseases § Accelerated Approval
- To speed approval in a life-threatening disease based on surrogate marker data with a commitment to conduct confirmatory studies § Key Consideration - Benefit risk assessment
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Drug Development Imperative: Prepara&on of Target Product Profile
§ Considerations – Indications – Target patient populations – Efficacy profile – Safety profile – Formulations – Dosage regimens
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Input Into Drug Development Program
§ Involve Constituencies
-‐ Regulatory Agencies
-‐ Key Investigators & Opinion Leaders (KOLs) -‐ Patient Advocacy Groups
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Clinical Trial Statistical Considerations Two Separate Assessments
Pre-‐Study Sample Size Determina3on
(Output: number of pa3ents)
Assump&ons
§ Power (80%) -‐ Probability of sufficient number of
patients studied to detect a difference (avoid false negative)
§ α value (≤0.05)
-‐ Probability of result occurring by chance (avoid false positive)
§ Difference to be detected
§ Enrollment period
§ Follow-‐up dura3on
[Prespecified Sta&s&cal Analysis Plan]
Post-‐Study Sta3s3cal Analysis
(Output: p value result)
Assessments
§ Primary endpoint (eg, pa3ent survival)
§ Sta3s3cal test to determine p value (eg, modified log-‐rank test to compare Kaplan-‐Meier pa3ent survival curves)
[Post Hoc Sensi&vity Analyses]
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Regulatory Meeting Preparation
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§ Back to Basics
• WHO • WHAT • WHERE • WHY • HOW
Regulatory Preparation: Back to Basics
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§ WHO
• Iden6fy individuals from the Regulatory Agencies who will be involved in interac6ons so the right company and external experts are available for mee6ngs and/or teleconferences
• Contact external experts as soon as possible to ensure their availability
(is going to be involved)
Regulatory Preparation: Back to Basics
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§ WHAT
• Compile a complete list of ac6vi6es that need to be accomplished for the mee6ng
• Do not overlook details which can cause delays or inconvenience such as booking a sufficient number of hotel rooms
(needs to be done)
Regulatory Preparation: Back to Basics
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§ WHERE
• Determine loca6on of mee6ngs early on to facilitate travel and hotel bookings
• Prepare for con6ngencies such as the possibility of extra aRendees
(will prep meetings and actual meeting be held)
Regulatory Preparation: Back to Basics
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§ WHY (the need for a mee6ng should be asked to provide clarity of mee6ng goals and details to facilitate prepara6on)
• Establish rapport as possible with Regulatory Agency staff to assist with planning
• Hold prep teleconferences and/or mee6ngs to minimize disconnects
Regulatory Preparation: Back to Basics
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§ HOW • Prepare a 6me and event schedule to delineate expecta6ons
(will logistics be coordinated to avoid duplication of activities)
Regulatory Preparation
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§ Needs
• Iden6fy the goals for the mee6ng • Understand how work is distributed • Foster collabora6on
Regulatory Preparation
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§ Details
• Know the audience -‐ Obtain informa6on on their exper6se
• Try to preempt an6cipated ques6ons in the briefing document -‐ Understand Agency stance on topic(s) to be discussed
Drug Development
§ Critical Success Factor − Upfront planning to prevent problems
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Drug Development
Quality
Time Cost
Drug Development
Align Goals and Incentives for Success
Key Factors in Drug Development - Balance
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